What Patient Population Is Participating In Your Early-Phase Gene Therapy Trials? These Factors Could Influence Your Strategy

By Kenneth Ndugga-Kabuye

Presently, in the United States, there exist over 10,000 rare diseases that affect in excess of 30 million individuals¹. Many of these conditions pose life-threatening risks, and dishearteningly, over 90% lack an approved treatment sanctioned by the U.S. FDA². However, a ray of hope shines through the realm of novel gene therapies, offering a promising avenue for enhancing the well-being of individuals grappling with these afflictions.

Nonetheless, the journey to secure regulatory clearance for these treatments is an intricate and multifaceted one. In an endeavor to facilitate productive dialogue with key stakeholders in product development and researchers traversing the approval pathway, the FDA's Office of Tissues and Advanced Therapies (OTAT) within the Center for Biologics Evaluation Research (CBER) has introduced a virtual Town Hall series. This initiative serves as a platform where participants can glean invaluable regulatory insights to advance their drug development efforts. Specifically, the February 7, 2023 session of the OTAT Town Hall series was dedicated to the critical subject of gene therapy clinical development for rare diseases.

While the February event encompassed a spectrum of vital points, this post serves to outline the discussion pertaining to the selection of patient populations in early-phase studies. Within this discourse, pertinent clarifications and insights were offered concerning the optimization of the benefit-risk profile, considerations related to adult versus pediatric participation and consent, the role of healthy volunteers, and the importance of customization in the clinical development process.